Clinical Research Alert: Clinical Study for Children and Teenagers with SMA

Researchers at argenx are conducting a phase 2 clinical trial (SPARKLE) for children and teenagers with spinal muscular atrophy (SMA). The study is designed to determine the appropriate dose of the investigational drug adimanebart (ARGX-119) and to evaluate its safety and effectiveness as a potential treatment for SMA. Researchers will also examine how the drug is processed by the body and how the immune system responds to it.

The study

This study will compare the effects of ARGX-119 with a placebo. During the 24-week main double-blinded period, participants will be randomly assigned to receive either the investigational study drug or a placebo control via intravenous (in the vein) infusion.

Before this phase, there will be a screening period of up to 4 weeks to determine eligibility. After the double-blinded period, participants may enter an active-treatment extension period of up to 100 weeks, during which all participants will receive the investigational study drug. A safety follow-up period of 20 weeks will take place after the last dose.

In total, participation in the study may last up to about three years and will include approximately 38 clinic visits over the course of the trial.

Study criteria

To be eligible, individuals must meet the following inclusion criteria:

• Between 5 and less than 18 years of age
• Diagnosed with 5q-SMA
• Able to walk at least 50 meters without walking aids
• Currently receiving a stable SMA treatment regimen (nusinersen or risdiplam) and/or have a history of onasemnogene abeparvovec treatment

Additional eligibility criteria may apply. For more details about this study, please visit this link.

Interested in participating?

Travel support is available for eligible participants and their families.

To learn more about the study or inquire about participation, please visit the sponsor’s trial website or contact the study coordinator by email: clinicaltrials@argenx.com.

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